CFQ-R questionnaire and CF candidates for lung transplant

International Study: “CFQ-R questionnaire and CF candidates for lung transplant”. First part of international project: A novel score for lung transplant indication in Cystic Fibrosis”

INTRODUCTION

Although there are international guidelines on when to transplant the Cystic Fibrosis (CF) population, the precise moment to place a CF patient on the list for lung transplantation (LT) remains uncertain. Nevertheless, hundreds of CF patients undergo LT every year. There are predictive models of survival that help determine the physiological readiness for transplantation in this population. Also, there is an international consensus, mainly based on respiratory functional parameters on when LT is indicated for patients with chronic respiratory diseases. In general, the main LT criteria for the CF population include: an FEV1 less than 30% predicted, a rapid decline in lung function, an increase in the number of exacerbations, progressive respiratory insufficiency, and/or life-threatening hemoptysis, among other factors. However, the FEV1 under 30% is not necessarily the best criterion for LT. In fact, there are CF patients with better lung mechanics that have other clinical parameters determining their need for lung transplantation. At the same time there are other CF patients with a lower FEV1 that have other stable clinical parameters whereby the risk of LT outweighs its benefit.

There are still patients who die on the waiting list which means, in part, that the indication for LT is not precisely defined. There is a need for precision in determining the proper time to list and more importantly when to refer a CF patient to a lung transplant center. In addition, there is a need to determine which factors will best predict mortality in this population.

According to the ISHLT 2006 guidelines, the decision for LT is derived from a comprehensive evaluation that takes into account several indicators of disease severity such as: FEV1, increases in oxygen need, hypercapnia, the need for noninvasive ventilation, functional status (e.g., 6-MWD), and pulmonary hypertension.

The current guidelines for referral are:

FEV1 below 30% predicted or a rapid decline in FEV1, particularly in young female patients.

Exacerbation of pulmonary disease requiring ICU stay.

Increasing frequency of exacerbations requiring antibiotic therapy.

Refractory and/or recurrent pneumothorax.

Recurrent hemoptysis not controlled by embolization.

However, there is not an established score to predict the optimal moment to list a CF patient for LT.

AIM

Recently a specific questionnaire on quality of life (CFR-Q) has been demonstrated to accurately detect variations in lung function and changes in clinical performance of CF patients. In fact, all recent trials in this population are including this questionnaire as an end point. The principal aim of this study is to apply this CFR-Q to all CF patients with an FEV1 below 40% to determine if this questionnaire can detect any significant changes in any of the domains that correlate with lung function and the variables that correlate with severe advanced disease.